FAST to Advance Gene Therapy Candidate through IND-Enabling Studies Conducted in Partnership with the University of Pennsylvania
The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS). Angelman syndrome is a rare neurogenetic disorder that is estimated to impact approximately […]
The National Institutes of Health awarded the first phase of a roughly $40M grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases
In October 2023, the National Institutes of Health awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4 (HIST1H1E) syndrome. The project team named under […]